Study of a pd VWF/FVIII Concentrate, Biostate®, in Subjects With Von Willebrand Disease

NCT00941616 | Completed Phase 2 DMC

• 3 other identifiers: CSLCT-BIO-08-5414812008-004922-18
• Study Type: interventional
• Target: 22
• Locations: 4 countries
• Sites: 5

Brief Summary

The aim of this study is to assess the pharmacokinetics (PK), efficacy, and safety of Biostate® in subjects with Von Willebrand Disease (VWD). Pharmacokinetic Component: PK parameters will be determined from a subgroup of subjects. Subjects who complete the PK component will subsequently continue in the efficacy component of the study, either continuing on a previously established prophylaxis regimen or continuing to receive on-demand treatment with the occurrence of non-surgical bleeding (NSB) events. Efficacy Component: Three treatment arms are defined for the efficacy component of the study. (1) Subjects who are currently being treated on a set prophylaxis regimen with a VWF product at the time of study entry will be enrolled in the "Prophylaxis" arm. (2) Subjects not being treated on a set prophylaxis regimen at the time of study entry who require a VWF product for the treatment of NSB events will be enrolled in the "On-demand" arm and commence using Biostate in the treatment of NSB events. (3) Subjects enrolled in the "On-demand" arm have the possibility to enter the "Cross-over to Prophylaxis" arm to receive an additional 12 months of prophylactic treatment.

Conditions

Von Willebrand Disease

Keywords

Von Willebrand Disease

Outcome Measures

Primary Outcomes (7)

• Haemostatic efficacy at time of non-surgical bleeding (NSB) event

  From Day 1 until final study visit

• Haemostatic efficacy overall

  Monthly (prophylactic therapy) or once every 3 months (for on-demand use)

• Number of treatments with blood product transfusions required to resolve any bleeding event

  From Day 1 until final study visit

• vWF/FVIII concentrate usage (number of infusions, IU/kg per dose, per event, per month and per year)

  From Day 1 until final study visit

• Assessment of blood loss during any surgical procedure

  From Day 1 until final study visit

• Number of spontaneous or traumatic NSB events

  From Day 1 until final study visit

• Pharmacokinetic parameters for vWF and FVIII (PK arm only)

  Up to 72 hours following infusions on Day 1 and approximately Day 180

Secondary Outcomes (2)

• Development of FVIII inhibitors

  From Day 1 until final study visit

• Development of vWF inhibitors

  From Day 1 until final study visit

Study Arms (4)

PK

EXPERIMENTAL

Includes subjects participating in the pharmacokinetic component of the study.

Biological: Biostate®

Prophylaxis

EXPERIMENTAL

Includes subjects receiving 12 months of prophylactic therapy.

Biological: Biostate®

On-demand

EXPERIMENTAL

Includes subjects receiving 12 months of on-demand treatment.

Biological: Biostate®

Cross-over to prophylaxis

EXPERIMENTAL

Includes subjects completing 12 months of on-demand treatment (the "On-demand" arm) who cross-over to prophylactic therapy for an additional 12-month period.

Biological: Biostate®

Interventions

Biostate® BIOLOGICAL

80 IU vWF/kg administered as a bolus intravenous infusion on Day 1 and approximately Day 180

Also known as: Human Coagulation Factor VIII / von Willebrand Factor

PK

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosed with VWD
• Desmopressin acetate (DDAVP) treatment is ineffective or contraindicated or not available
• Evidence of vaccination against hepatitis A and B (or presence of antibodies against hepatitis A and B) within 10 years prior to their first dose of Biostate®
• Written informed consent given

You may not qualify if:

• Actively bleeding immediately prior to initial PK period
• Have received DDAVP or a VWF product in the 5 days prior to their first dose of study product
• Have Type 2B, 2N or 2M VWD
• Requiring a VWF product for a planned surgical procedure at enrolment
• Have received aspirin or other non-steroidal anti-inflammatory drugs within 7 days prior to their first dose of study product
• Known history of, or are suspected to have, VWF or FVIII inhibitors
• Suffering an acute or chronic medical condition, other than VWD, which may affect the conduct of the study
• Known or suspected hypersensitivity or previous evidence of severe side effects to Biostate®, VWF/FVIII concentrates, or human albumin
• Impaired liver function at screening
• Evidence or a history (within the previous 12 months) of abuse of any drug substance, licit or illicit
• Participation in a clinical study or use of an investigational compound in the 3 months preceding the first day of study drug administration, or plans to enter such a study during the study period.
• Females who are pregnant, breast-feeding or who have a positive pregnancy test at screening

Sponsors & Collaborators

• CSL Behring: lead
• Parexel: collaborator

Study Sites (5)

Study Site

Sofia, Bulgaria

Location

Study Site

Warsaw, Poland

Location

Study Site

Wroclaw, Poland

Location

Study Site

Barnaul, Russia

Location

Study Site

Lviv, 79044, Ukraine

Location

MeSH Terms

Conditions

von Willebrand Diseases

Interventions

Factor VIII; von Willebrand Factor

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Blood Platelet Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Study Officials

• Program Director, Clinical R&D

  CSL Behring

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 15, 2009
• First Posted: July 17, 2009
• Study Start: June 1, 2009
• Primary Completion: February 1, 2012
• Study Completion: February 1, 2012
• Last Updated: October 3, 2017

Record last verified: 2017-10

Locations

UA (1); RU (1); PL (2); BU (1)